Loss of TDP-43 nuclear function is a major event in ALS pathogenesis and leads to widespread RNA processing alterations including reduced stathmin-2 levels. Stathmin-2 is shown to be crucial for axonal regeneration and to represent a novel therapeutic target in ALS.

Massachusetts General Hospital investigators assessed the role of nuclear import receptor (NIRs) in disrupting such aggregates associated with neurodegenerative disorders, chaperoning these proteins from the cytoplasm to the nucleus and restoring their normal function, thereby rescuing the cell from degeneration.

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